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Enlarge ImageRequest to buy this photoEd Cunicelli | The Children’s Hospital of PhiladelphiaDr. Stephan A. Grupp checked Emily Whitehead at the Children’s Hospital of Philadelphia in October 2012.

In one of the biggest advances against leukemia and other blood cancers in many years, doctors
are reporting unprecedented success by using gene therapy to transform patients’ blood cells into
soldiers that seek and destroy cancer.

A few patients with one type of leukemia were given this one-time, experimental therapy several
years ago, and some remain cancer-free.

Now, at least six research groups have treated more than 120 patients with many types of blood
and bone-marrow cancers, with stunning results.

“It’s really exciting,” said Dr. Janis Abkowitz, blood-diseases chief at the University of
Washington in Seattle and president of the American Society of Hematology. “You can take a cell
that belongs to a patient and engineer it to be an attack cell.”

In one study, all five adults and 19 of 22 children with acute lymphocytic leukemia had a
complete remission, meaning no cancer could be found after treatment. A few have relapsed since
then.

These were gravely ill patients out of options. Some had tried multiple bone-marrow transplants
and as many as types of chemotherapy or other treatments.

Cancer was so advanced in 8-year-old Emily Whitehead of Philipsburg, Pa., that doctors said her
major organs would fail within days. She was the first child given the gene therapy, and nearly two
years later, she shows no sign of cancer.

Results on other patients with myeloma, lymphoma and chronic lymphocytic leukemia will be
reported at the hematology group’s conference that started yesterday in New Orleans.

Doctors say this has the potential to become the first gene therapy approved in the United
States and the first for cancer worldwide. Only one gene therapy is approved in Europe, for a rare
metabolic disease.

The treatment involves filtering patients’ blood to remove millions of white blood cells called
T-cells, altering them in the lab to contain a gene that targets cancer, and returning them to the
patient in infusions over three days.

“What we are giving essentially is a living drug” — permanently altered cells that multiply in
the body into an army to fight the cancer, said Dr. David Porter, a University of Pennsylvania
scientist who led one study.

Several drug and biotech companies are developing these therapies. Penn has patented its method
and licensed it to Switzerland-based Novartis AG. The company is building a research center on the
Penn campus in Philadelphia and plans a clinical trial next year that could lead to federal
approval of the treatment as soon as 2016.

Nearly 49,000 new cases of leukemia, 70,000 cases of non-Hodgkin lymphoma and 22,000 cases of
myeloma are expected to be diagnosed in the United States in 2013.

Many patients are successfully treated with chemotherapy or bone-marrow or stem-cell
transplants, but transplants are risky, and donors can’t always be found.

So far, gene therapy has been tried on people who were in danger of dying because other
treatments failed.